{"id":"W7133021897","doi":"","title":"Spell Checking Nature: Development of a CRISPR- Mediated Gene Editing Approach for the Treatment of Pathogenic Duplications","year":2020,"lang":"","type":"dissertation","venue":"TSpace","topic":"Muscle Physiology and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":0,"is_retracted":false,"has_abstract":true,"ca_institutions":"Amgen (Canada)","funders":"Muscular Dystrophy Canada; Hospital for Sick Children; Duchenne Research Fund","keywords":"Gene duplication; Muscular dystrophy; Dystrophin; Duchenne muscular dystrophy; Exon; Gene; Genome editing; Tandem exon duplication; CRISPR","routes":{"ca_aff":true,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":false},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.000306605,0.0006768932,0.0004816546,0.0006547279,0.0003238194,0.0006041534,0.0007000113,0.001114836,0.004147902],"category_scores_gemma":[0.0002344899,0.0003219331,0.0006525935,0.00022261,0.0002944974,0.0004109216,0.0005041595,0.001383281,0.001756771],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004255737,"about_ca_system_score_gemma":0.0004822863,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001126189,"about_ca_topic_score_gemma":0.002030102,"domain_scores_codex":[0.9996855,0.00002650146,0.00001570517,0.00007139028,0.0001611747,0.00003976265],"domain_scores_gemma":[0.9999003,0.00001204449,0.00002150001,0.000008995806,0.00002578161,0.00003142592],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"bench_or_experimental","study_design_scores_codex":[0.0001622888,0.0001586056,0.0002232971,0.0002813648,0.00004498996,0.0005548527,0.00004696248,0.000880436,0.916721,0.001727549,0.005165169,0.07403338],"study_design_scores_gemma":[0.0002160402,0.001290959,0.001951808,0.000083862,0.0001251175,0.004344888,0.00003533914,0.008615306,0.8575566,0.0009501568,0.1247519,0.00007803339],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"methods","genre_gemma":"empirical","genre_scores_codex":[0.3091136,0.04938523,0.5369908,0.007874928,0.00439973,0.001874795,0.00454141,0.0120836,0.07373581],"genre_scores_gemma":[0.6763378,0.02785033,0.1996027,0.003488607,0.000285525,0.0008985,0.003168444,0.0009274376,0.08744074],"genre_candidate":"empirical","genre_consensus":null,"teacher_disagreement_score":0.004147902,"threshold_uncertainty_score":0.01387614,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02096938202636429,"score_gpt":0.319798531339972,"score_spread":0.2988291493136077,"validation_status":"score_only:v0-immature-baseline","note":"Baseline scores from an immature model (maturity gate not passed). Scores rank; they never assert a category."}}